Tuesday, January 10, 2017

Cancer 'wonder drug' given tick of approval

In a major milestone for Melbourne scientists, a leukaemia drug almost 30 years in the making has been given the governmental tick of approval.

It’s touted as a wonder drug that "melts away" cancer cells, but patient John Higham would go one step further.

“To me, it's a miracle. I mean, I'm agnostic, but to me, it's a miracle. My life's been saved by this drug,” he told SBS News.

The 65-year-old was diagnosed with Chronic Lymphocytic Leukaemia, or CLL, in 2007. Two rounds of chemotherapy helped, but didn't rid him of the advanced blood cancer.

Three years ago, doctors put him into the Venetoclax drug trial, a last-resort treatment reserved for patients who were not responding to traditional therapy.

Laboratory head at the Walter and Eliza Hall Institute of Medical Research, Professor David Huang, told SBS the drug works by targeting the over-active protein known as BCL-2, which enables cancer cells to grow.

“Essentially it's designed to trigger the cancer cells to commit cell-suicide,” he said.

Haematologist at the Royal Melbourne Hospital and Victorian Comprehensive Cancer Centre, Dr Mary Ann Anderson, said the effectiveness of the drug exceeded all expectations.

“The first patient in the world, had a football [sized tumour], underneath his arm. Within a week, it was a golf ball, and within a month, we couldn't feel anything.”


The hard-to-treat cancer affects about 1300 Australians a year.
About 100 patients have taken part in clinical trials, which began in 2013.

Eighty per cent of patients responded, with 20 per cent now in full remission.

“Their lymph nodes shrunk away, they became transfusion independent, and their symptoms settled down,” Dr Anderson said.
“For other patients, just having control of the disease is a huge weight off their mind.”

TGA Approval

Following the success of the trials, Venetoclax has now been granted approval by the Therapeutic Goods Administration (TGA).
It was approved for use in the US in April last year, and in December, the EU approved it too.

It's a milestone for the Melbourne researchers who've been developing the drug for over 28 years.

“As a basic scientist, what you dream of, is that what you do, and your work on the bench, will make a difference for patients,” said Professor Huang.

The next step is having Venetoclax listed on the pharmaceutical benefits scheme (PBS), subsidising it so that it's not cost-prohibitive for patients.

That process is already underway, and a decision could be made in the next few months.

While John Higham will need to continue taking three pills a day despite now being cancer-free.

But for him, it's a small price for his quality of life.

“I call myself cured,” Mr Higham said.

“They say I'm in remission, but I call myself cured.”

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